Title: FDA FVIII vCJD Risk Assessment: A global perspective
1FDA FVIII vCJD Risk AssessmentA global
perspective
Mark W. SkinnerWFH President
- 15 December 2006 FDA TSEAC Meeting
Washington, DC
2WFH Guidance on Assessing Product Risk
- It is important to avoid the complacency which
characterized previous blood borne epidemics in
people with hemophilia, while retaining a sense
of proportion to this particular issue, and
maintain a constant watch on the balance between
safety and supply - Structured risk-management processes require a
strategy for continuous learning and communication
A. Farrugia vCJD and Hemophilia Further
Guidance on Assessing the Risk of Plasma-Derived
Products for Treating Hemophilia WFH 2004
3Risk Communication Core Values
- Follow a precautionary approach when scientific
knowledge is incomplete - Patients have a right to be consulted and
informed - Timely communication with openness and
transparency - Clear and comprehensible explanations essential
- Appropriate to acknowledge areas of uncertainty
- Avoid unintended health consequences
- e.g.) patient stigmatization, denial of access to
care
4Potential Global Implications
- 75 of the patients in the world receive little
or no care - Outside of the highly developed nations,
treatment products are predominantly
plasma-derived
Source WFH Global Survey 2005 Random sample of
45 countries
5Placing Risk in Context
- Mortality and Causes of Death in PWH
- Before the introduction of clotting factor
preparations, the mean life expectancy of
patients with hemophilia was lt30 years, and
patients mostly died of intracranial or other
hemorrhages - In patients with severe hemophilia not infected
with viruses, mortality is still 40 higher when
compared with the general population - 27 (13/49) of the deaths in the cohort of
patients not infected with HCV or HIV were
because of hemorrhage
PLUG, I., et. al., Mortality and causes of death
in patients with hemophilia, 19922001 a
prospective cohort study. Journal of Thrombosis
and Haemostasis 4 (3), 510-516. doi
10.1111/j.1538-7836.2006.01808.x
6Quantifying the Risk in Global Context
- FDA risk assessment builds on global knowledge
and understanding - U.K. 2004 U.K. plasma-derived concentrates 1
additional risk beyond general population risk of
14,225 to 1.81,000,000 - FDA 2006 U.S. plasma-derived concentrates
between 1105,000 19,400,000 - FDA assessment is significantly lower than the
U.K. assessment and thus provides further
reassurance for patients clinicians
7Selection of Treatment Product
- The FDA assessment adds to our understanding and
provides reassurance for both clinicians and
patients when making treatment product selection
decisions - The WFH continues to view both recombinant and
plasma-derived products as important treatment
options for the global bleeding disorders
community - Potential remains for adverse events including
inhibitor development or unknown pathogen risk,
thus continuous learning and communication are
required